New Facility Dedication Unveils Unique Environment for Research Breakthroughs at The Children's Hospital of Philadelphia

--In Colket Building, Scientists Will Translate Research into Real-Life Treatments for Children --

PHILADELPHIA, June 9 /PRNewswire-USNewswire/ -- The Children's Hospital of Philadelphia will dedicate its new Ruth and Tristram Colket, Jr. Translational Research Building on Wednesday, June 9, at 11 a.m. Launched with an initial gift of $25 million from Ruth M. and Tristram C. Colket, Jr., the 12-story, environmentally friendly structure houses high-profile, cutting-edge programs for advancing research in pediatric diseases, with additional programs scheduled to move in over the coming months.

"This facility provides a new, custom-designed setting to advance our already robust research to the next level," said Stephen B. Burke, chairman of the Board of Trustees of Children's Hospital. "We are building more than glass and steel; we are building 21st century health care for children worldwide."

An initial $25 million contribution by long-time board members Ruth M. and Tristram C. Colket, Jr. helped to launch construction more than four years ago. "Children in the Philadelphia area and around the world have benefited greatly from the generous support provided by Ruth and Tristram Colket over the past 40 years," said Steven M. Altschuler, M.D., the Hospital's president and chief executive officer. "This new building bearing their names honors their long-standing commitment to the health of children."

The $504 million project encompasses 700,000 square feet—four new laboratory floors, administration and conference space, and a two-story ground floor housing a lobby and cafeteria. There are an additional four stories below grade consisting of infrastructure and laboratory support space.

The Colket Translational Research Building will enable scientists across a variety of disciplines, all dedicated to developing treatments for a specific pediatric disease, to work side-by-side.  Physical proximity will foster close collaboration, thus providing more opportunities for new ideas and expedited results. In keeping with the building's research focus, many of the current floors feature state-of-the-art laboratories, which can be reconfigured easily as teams grow, and their research evolves.  The building will expand along with Children's Hospital's research program; it can accommodate 12 additional floors to reach 24 stories.  

"Translational research entails transforming scientific discoveries into medical innovations," said Philip R. Johnson, M.D., chief scientific officer at Children's Hospital. "Those innovations are aimed at improving the lives of children and families. Having state-of-the-art space, equipment and technology will help us attract top-level researchers to join the preeminent scientists already here."

Pacesetting Research in Pediatric Cancer, Mitochondrial Disorders and Gene Therapy

The third and fourth floors of the CTRB are home to one such collection of preeminent scientists, at the Center for Childhood Cancer Research. One of that program's research interests offers an outstanding example of expediting the bench-to-bedside process: oncology researchers moved from announcing the discovery of a gene variant for neuroblastoma, a childhood cancer of the peripheral nervous system, to opening a clinical trial based on that discovery, in only 12 months—an uncommonly rapid pace in translational cancer research. Yael P. Mosse, M.D., and John M. Maris, M.D., the Center's director, reported in August 2008 that mutations in the ALK gene give rise to some forms of neuroblastoma. Within a year, the researchers were enrolling their first patients in a clinical trial of an ALK inhibitor, a drug that blocks a protein on a biological pathway that leads to neuroblastoma. While it was fortuitous that the ALK inhibitor had already been developed for testing other forms of cancer, the trial also rested on a decades-long history of neuroblastoma research at Children's Hospital and on the Hospital's well-oiled, established infrastructure for supporting clinical trials.

Another approach used by pediatric oncologists at the Cancer Center uses immunotherapy, which harnesses elements of the body's immune system to attack cancer cells. One current treatment, part of a national, multicenter trial that has boosted the survival rate in neuroblastoma, uses genetically engineered proteins (cytokines and monoclonal antibodies) to zero in on neuroblastoma tumors. Stephan A. Grupp, M.D., Ph.D., a researcher in this trial, is refining this approach with techniques that rely on cancer fighting T-cells from each child patient rather than on proteins. Grupp and colleagues at Penn Medicine will modify those T-cells by attaching a molecule tailored to direct the T-cells to target the cancer cells, then will create large numbers of those engineered cells and return them to the patient. The hope is that this highly specific approach will be more effective than current treatments, and gentler on children, by not necessitating additional drugs with harsh side effects. He expects to start a clinical trial of this T-cell immunotherapy soon against leukemia, with a trial to follow against neuroblastoma.

Another entire floor of the building will be dedicated to researchers investigating mitochondria, the tiny power plants of human cells. When mitochondria do not function properly, they lead to a large number of metabolic and degenerative diseases, and also play an important role in diabetes, epilepsy, Parkinson's disease and Alzheimer's disease. Douglas C. Wallace, Ph.D., one of the world's leading experts in mitochondria, is bringing his laboratory to Children's Hospital to establish a Center for Mitochondrial and Epigenomic Medicine. More than 35 years ago, Wallace was a founder of mitochondrial genetics, elucidating how changes in mitochondrial DNA reveal human origins and early migrations. He showed that mitochondrial DNA is inherited exclusively from mothers, and collaborated on work describing the evidence for "mitochondrial Eve," the female ancestor of humanity who lived in Africa about 200,000 years ago. His program at CHOP will include basic research, diagnostics and clinical applications.

On another floor of the building, research staff members formerly spread out over several sites now have a central location for their efforts in two emerging fields of medicine—gene therapy and stem cell therapy. At the Center for Cellular and Molecular Therapeutics, directed by internationally recognized gene therapy pioneer Katherine A. High, M.D., a new $2 million NIH grant from federal National Recovery Act funds is enabling the expansion and upgrade of a manufacturing suite at the Clinical Vector Core facility. This facility produces clinical-grade vectors—bio-engineered viruses that deliver therapeutic genes to patients suffering from genetic diseases.

It supplied vectors used in one of the most dramatic medical successes of 2009—the gene therapy trial for an inherited form of blindness called Leber's congenital amaurosis. National news broadcasts featured nine-year-old Corey Haas, whose vision has improved to the point that he stopped using his cane, can see a blackboard, and can ride a bicycle unaided. Under its national contract, the upgraded facility will produce vectors for NIH-supported scientists investigating a multitude of genetic disorders at Children's Hospital, the University of Pennsylvania, and at many other centers. Within the next year, High's center expects to supply vectors for a new clinical trial of gene therapy for the inherited bleeding disorder hemophilia.

Embedded in the Hospital's Research Matrix

As impressive as the research programs filling up the CTRB will be, they are components of a broader campus-wide research enterprise, under the administrative umbrella of The Children's Hospital of Philadelphia Research Institute. The Institute includes major centers and research affinity groups, which bring together scientists from multiple disciplines in fruitful interactions and collaborations. One such program is the Center for Autism Research, a comprehensive, premier program in autism spectrum disorders, which is engaged in groundbreaking imaging studies of brain mechanisms involved in autism, as well as early risk studies and the world's largest randomized study of behavioral treatments for autism, to name just a few projects.

Other research highlights include the Center for Applied Genomics, a world-class program in pediatric genetics research, which has discovered genes involved in asthma, autism, diabetes, inflammatory bowel disease, obesity, schizophrenia, neuroblastoma and ADHD; the Center for Pediatric Clinical Effectiveness, aimed at discovering and disseminating best practices in preventing and managing childhood diseases, for clinicians on the front lines of pediatric healthcare; and the Hospital's deep and distinguished record of research in childhood vaccines. Taking a notable recent example, vaccine research here led to a rotavirus vaccine that is saving hundreds of thousands of lives around the world.

"We have a great deal to be proud of when looking at our Hospital's global impact," concluded Johnson. "Bringing research leaders together in this new facility enhances both our capabilities and our opportunities to transform pediatric medicine."

Located on Civic Center Boulevard on an eight-acre site directly across from the Hospital's main clinical and research facilities, the Colket Translational Research Building (CTRB) had its groundbreaking in June 2006.

The CTRB has a Silver LEED certification, reflecting its energy-saving, environmentally responsible design features, such as heat-reflective roofing and windows, energy recovery systems in heating and air conditioning, light harvesting system and low-flow plumbing.

About The Children's Hospital of Philadelphia: The Children's Hospital of Philadelphia was founded in 1855 as the nation's first pediatric hospital. Through its long-standing commitment to providing exceptional patient care, training new generations of pediatric healthcare professionals and pioneering major research initiatives, Children's Hospital has fostered many discoveries that have benefited children worldwide. Its pediatric research program is among the largest in the country, ranking third in National Institutes of Health funding. In addition, its unique family-centered care and public service programs have brought the 460-bed hospital recognition as a leading advocate for children and adolescents. For more information, visit http://www.chop.edu.

SOURCE The Children's Hospital of Philadelphia